Anti-Infective Drug Products for the Pediatric Population – Guidance from FDA

The U.S. Food and Drug Administration (FDA) has issued final guidance to support sponsors in developing anti-infective drug products—including antibacterials, antifungals, and antiparasitic agents—for pediatric patients. The guidance outlines key considerations for efficacy, study design, safety, and cohort selection during pediatric drug development.

Drug Development Considerations

(1) Efficacy Extrapolation

(1.1) Efficacy from well-controlled adult clinical trials may be extrapolated to pediatric populations when: 

(1.1.1) The disease behaves similarly in adults and children, including comparable disease processes and causative organisms. 

(1.1.2) The drug product’s therapeutic effect is sufficiently similar across adult and pediatric populations.

(1.2) When disease characteristics differ between adult and pediatric groups—or among pediatric subgroups—extrapolation may not be appropriate. 

(1.2.1) In such cases, independent, well‑controlled pediatric clinical trials may be required to support the indication.

(2) Cohorts Based on Age, Body Weight, or Body Surface Area

(2.1) Phase 3 trials in adults should include adolescents (12 years and older) once sufficient adult safety data exist to evaluate risks and potential benefits. FDA strongly encourages adolescent enrollment when scientifically and ethically appropriate.

(2.2) Pediatric cohorts should be determined based on disease incidence and drug‑specific considerations such as pharmacokinetics or safety characteristics. Parallel enrollment of multiple age groups is encouraged when no safety or PK concerns necessitate sequential enrollment.

(2.3) Neonates present unique challenges due to dosing complexities required to achieve effective and safe drug exposure.

(2.4) Sponsors should assess whether obesity affects dose selection.

(3) Safety Data

(3.1) Safety data should be collected using the intended pediatric dose and treatment duration.

(3.2) For anti‑infective drugs developed for a pediatric‑specific indication, FDA recommends including available supportive safety and efficacy data from related adult indications (e.g., acute bacterial sinusitis). Development plans should be discussed with FDA.

(3.3) Safety data from nonclinical studies, class‑wide safety concerns, and adult safety profiles can provide context and identify adverse events for pediatric evaluation. However, toxicities affecting developing organs may require additional assessments, particularly in younger age groups.

(3.4) The recommended pediatric safety database size depends on factors such as disease incidence, known or anticipated adverse events, and expected drug use in children. Sponsors should discuss database size with FDA as clinical development progresses.

(3.5) Pediatric anti‑infective trials generally use an active comparator reflecting the standard of care at study sites. Unequal randomization favoring the investigational arm is acceptable.

(3.6) Comparative pediatric safety data may be needed for new drug classes or products with concerning nonclinical safety findings.

(3.7) Non‑comparative studies may be acceptable when the drug belongs to a class with a well‑established safety profile in both adults and children.

(3.8) When dosage and duration are consistent across multiple indications, safety data from one indication may be used to support safety for others.